Achondroplasia Research

Did you know that #achondroplasia gets its name from Greek, meaning "without cartilage growth"? Caused by a change in the FGFR3 gene, achondroplasia is the most common type of skeletal dysplasia, occurring in about one in 25,000 people. Although there are some medical considerations, many people with achondroplasia can live healthy, fulfilling, and purposeful lives. Learn more on MyAchonJourney: myachonjourney.com/achondroplasia…

Re: @BridgeBioPharma's oral #infigratinib for #achondroplasia: "This is a really promising medicine. If you're teaching trainees about achondroplasia, please don't forget to mention the non-surgical options." – @BSCOS_UK PODcast Listen here (Spotify): open.spotify.com/episode/4jGQYx…

Phillipe Backeljauw features in our upcoming 'Beyond Achondroplasia' workshop! 📢 He will address 'Treating Noonan, Turner, and SHOX' on the 29th of April from 17:30 BST. Register here: 🔗ow.ly/xPm650Vvtrp

We had a great time participating at the @eposorg Annual Meeting, connecting with advocates and scientific leaders in the community. Thank you to those who tuned in as we shared findings from our PROPEL 2 clinical study for children living with #achondroplasia. #EPOS2025

Now Enrolling: BEACH301 Clinical Trial In partnership with Tyra Biosciences, Rare Disease Research (RDR) is committed to advancing research and providing access to a new clinical trial for eligible children with achondroplasia. Learn more at achondroplasia.bio

We are delighted to announce the publication of our latest manuscript, ‘Recommendations for management of infants and young children with achondroplasia: Does clinical practice align?’ 🎉 You can read it in full here: 🔗ow.ly/7uVL50Vth0F

.@AscendisPharma Submits U.S. NDA for #TransConCNP (#Navepegritide) for the Treatment of Children with #Achondroplasia thechandlerproject.org/2025/04/01/asc…

#AbbiskoTherapeutics Announces IND Clearance from the Center for Drug Evaluation (CDE) of the China National Medical Products Administration for #ABSK061, a Highly Selective FGFR2/3 Inhibitor, for the Treatment of #Achondroplasia thechandlerproject.org/2025/03/27/abb…

Connecting with other families living with achondroplasia can be an invaluable source of support. On MyAchonJourney, you’ll find personal insights and perspectives from the community, as well as information on advocacy organizations and programs that help foster meaningful connections. Explore more: #building-a-support-network" target="_blank" rel="nofollow noopener">myachonjourney.com/achondroplasia…

We are excited to partner with @tyrabiosciences Biosciences to bring the BEACH301 clinical trial for eligible children with achondroplasia to families in the southeastern US, starting in Atlanta. 🌐 Visit at achondroplasia.bio to learn more. #Achondroplasia

Interested in exploring advancements in achondroplasia and related conditions? Join the first IAF Workshop 'Beyond Achondroplasia' workshop on the 29th of April from 17:30 BST. Register here 👉 ow.ly/fP7K50VoMo8

Today on #RareDiseaseDay, we're proud to join the #raredisease community to raise awareness about the millions of people around the world living with rare conditions. We recognize and celebrate the resilience of individuals living with rare conditions and their loved ones.

We're pleased to announce that positive 18-month results from PROPEL 2, a Phase 2 trial of the investigational therapy infigratinib in children with #achondroplasia, were published as an original research article in the New England Journal of Medicine (NEJM) today. Read more here: bit.ly/3YWTSMz

A promising daily tablet is effective at increasing height and improving proportional limb growth in children with #achondroplasia, the most common form of #dwarfism, a new #MCRI led study shows. #MedicalResearch #ChildHealth @RaviSavarirayan ➡️ direc.to/mXG6

Original Article: Oral Infigratinib Therapy in Children with Achondroplasia (PROPEL2) nej.md/3Z4xCSe #ESPE2024 | @EuroSPE

A study has determined that most children with #achondroplasia have sleep disordered breathing, and that its treatment is not always effective and that follow-up cardiorespiratory studies are typically required. Read more: bit.ly/3ZaeH8C #Pulmonology

We’re thrilled to welcome Dr. Shawn Standard to Nemours Children’s Hospital, Florida! Dr. Standard joins our team to expand the Limb Lengthening & Reconstruction Program alongside Dr. Jason Malone, offering care to children with limb deformities and orthopedic trauma.

At the International Skeletal Dysplasia Society Meeting 2024, Dr. @RaviSavarirayan shared updates from our PROPEL 2 program for achondroplasia and our ACCEL program for hypochondroplasia. Learn more: bit.ly/3ZtVgIe #ISDS2024

Data examining the effect of treatment on #BoneStrength in children with #achondroplasia will be presented at #ISDS2024. Learn more: bit.ly/4etglXs. This post is intended for US healthcare professionals only.

Today is the day, our first-ever poster is available to view NOW at #ISDS2024! 📃 You can also view our poster on “Practical Considerations for Following Adults with Achondroplasia – A Patient-held Checklist," (C-0023) as part of ‘Poster Session 1’ happening from 15:50 - 16:50.